<h2 dir="ltr">Mucopolysaccharidosis Type I (MPS I)</h2>
Mucopolysaccharidosis Type I (MPS I) is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase (IDUA), resulting in the accumulation of glycosaminoglycans (GAGs), including dermatan sulfate and heparan sulfate, in multiple organs and tissues. Progressive GAG accumulation leads to multisystem complications affecting skeletal, cardiac, respiratory, ocular, and neurological function. Early diagnosis and timely intervention are critical to improving long-term outcomes.
<h2 dir="ltr">Epidemiology Segmentation</h2>
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Diagnosed Prevalent Cases of MPS I
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Severity-specific Diagnosed Prevalent Cases of MPS I
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Total Treated Cases of MPS I
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<h3 dir="ltr"><a href="https://www.delveinsight.com/report-store/mucopolysaccharidosis-i-epidemiology-forecast">Mucopolysaccharidosis Type I Epidemiology</a></h3>
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Across the 7 Major Markets (7MM), the diagnosed prevalent population of MPS I was estimated at approximately 650 cases in 2025.
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The EU4 and the UK accounted for 62% of diagnosed prevalent cases, followed by the United States (37%) and Japan (1%).
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Among severity-specific diagnosed prevalent cases in the United States, Hurler Syndrome represented 57%, followed by Hurler-Scheie Syndrome (23%) and Scheie Syndrome (20%).
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The rarity of MPS I and variability in clinical presentation continue to contribute to underdiagnosis and delayed diagnosis, particularly among patients with attenuated disease forms.
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Improved awareness, advancements in genetic testing, and expansion of newborn screening programs are expected to enhance early detection and diagnosis rates during the forecast period.
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<h3 dir="ltr"><a href="https://www.delveinsight.com/report-store/mucopolysaccharidosis-i-market">Mucopolysaccharidosis Type I Market</a></h3>
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Within the 7 Major Markets (7MM), the United States captured the largest share of the MPS I market in 2025, accounting for approximately 50% of the total market.
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The MPS I market size across the 7MM was estimated at approximately USD 150 million in 2025.
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The market is projected to grow at a CAGR of 8.6% during the forecast period (2026&ndash;2036).
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Market growth is expected to be supported by earlier diagnosis, increasing utilization of enzyme replacement therapy, expanding newborn screening initiatives, and the advancement of innovative gene and cell-based therapies.
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<h3 dir="ltr">Mucopolysaccharidosis Type I Market Drivers</h3>
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Rising awareness among healthcare professionals and patients is supporting earlier diagnosis and treatment initiation.
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Expansion of newborn screening programs is improving identification of affected infants before irreversible disease progression occurs.
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Increasing adoption of enzyme replacement therapy continues to support market growth and improve patient outcomes.
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Growing research and development activities focused on gene therapy and next-generation treatments are expanding the future treatment landscape.
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<h3 dir="ltr">Mucopolysaccharidosis Type I Market Barriers</h3>
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Delayed and inaccurate diagnosis remains a major challenge due to the heterogeneous presentation of MPS I and overlap with other pediatric disorders.
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Current treatment options have limited effectiveness in addressing neurological manifestations and central nervous system involvement.
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Bone and joint complications continue to significantly impact quality of life despite available therapies.
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Limited access to specialized treatment centers and multidisciplinary care remains a challenge in several regions.
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<h3 dir="ltr">Mucopolysaccharidosis Type I Key Therapies</h3>
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OTL-203
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Lepunafusp alfa (JR-171)
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DNL622 (ETV:IDUA)
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ISP-001
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And others
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<h3 dir="ltr"><a href="https://www.delveinsight.com/report-store/mucopolysaccharidosis-type-i-pipeline-insight">Mucopolysaccharidosis Type I Key Players</a></h3>
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Orchard Therapeutics
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Kyowa Kirin
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JCR Pharmaceuticals
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Denali Therapeutics
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Immusoft
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BioMarin Pharmaceutical
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Sanofi
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And others.
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Mucopolysaccharidosis I Market

Mucopolysaccharidosis I Market Infographics

Mucopolysaccharidosis I Symptoms, Treatment, epidemiology, companies, drugs, infographic is a short guide to understand the problem and market landscape